The Michael J. Fox Foundation and The Silverstein Foundation for Parkinson's with GBA Grant Nearly $3 Million to Studies into Disease's Most Common Known Genetic Contributor
NEW YORK, Feb. 20, 2019 /PRNewswire/ -- The The Michael J. Fox Foundation for Parkinson's Research (MJFF) and The Silverstein Foundation for Parkinson's with GBA announce nearly $3 million in grants to studies investigating glucocerebrosidase beta acid (GBA). Mutations in the GBA gene are the most common genetic risk factors for Parkinson's, affecting about 10 percent of the more than 6 million people estimated to have the disease. The projects selected through this joint funding program aim to better understand the effect of GBA mutations — and the role of GBA more generally — and advance treatments against this target.
"Defining the GBA pathway and its role in disease, including in patients without a GBA mutation, could point to new therapeutic approaches that may slow or stop Parkinson's," said MJFF CEO Todd Sherer, PhD. "This partnership with the Silverstein Foundation streamlined the grant process to more quickly direct funding to these promising projects, speeding their efforts to help Parkinson's patients."
Silverstein Foundation Founder Jonathan Silverstein said, "We are very pleased with the collaboration with The Michael J. Fox Foundation and feel confident that the projects chosen will significantly add to the library of knowledge around GBA and propel new treatments for people living with Parkinson's and, perhaps, individuals at risk for the disease."
Previous research found that GBA mutations hamper activity of the glucocerebrosidase (GCase) enzyme, a member of the cell's cleaning crew that degrades damaged or excess cell parts. Build-up of cell parts (e.g., lipids and cellular proteins) can be toxic, resulting in the cell damage seen in Parkinson's disease. While linked directly to GBA gene mutations, GCase inhibition is also found in Parkinson's patients without these mutations. Therefore, therapies to heighten the enzyme's activities or mimic its effects may help a wider Parkinson's patient base. Three such medications are already in clinical trials.
From the 92 proposals submitted, MJFF and the Silverstein Foundation have selected 16 of the most promising projects to support through their joint funding program. Descriptions of the programs follow.
These projects aim to define novel biomarker candidates or therapeutic targets by investigating the role of GCase and impact of GBA mutations.
Two projects are looking for objective measures that could help in patient care and in research, by aiding in subject selection and therapeutic impact assessment.
Grantees are taking varied approaches to correct or counteract the effects of lower GCase activity.
About The Michael J. Fox Foundation for Parkinson's Research
As the world's largest nonprofit funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding more than $800 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson's research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson's disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson's awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world.
For more information, visit www.michaeljfox.org.
About The Silverstein Foundation for Parkinson's with GBA
The Silverstein Foundation is a 501(c)(3) non-profit organization focused on investing in cutting-edge therapeutic approaches for the treatment and prevention of Parkinson's disease in glucocerebrosidase (GBA) mutation carriers. The Foundation collaborates with clinicians, scientists, and biotechnology companies to accelerate research and clinical trials in an effort to rapidly bring new disease-modifying therapeutic options to patients. Under the leadership of a world-class team with strong domain expertise across neurodegenerative diseases, drug formulation chemistry, translational research, and drug development and commercialization, the Foundation has funded over 30 projects across seven different therapeutic approaches since its inception in 2017. The Foundation applies a unique flexible funding model including both new company formation and traditional research grants to deliver in real-time on its mission of rapidly developing novel disease-modifying treatments for Parkinson's disease.
For more information, visit www.silversteinfoundation.org.
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SOURCE The Michael J. Fox Foundation for Parkinson's Research